Evidence map›Paper›PMID 33283485›Full record

Trial reportESC heart failure2021

Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data.

Nicholas J Viney, Shuling Guo, Li-Jung Tai, Brenda F Baker, Mariam Aghajan, Shiangtung W Jung, Rosie Z Yu, Sheri Booten, Heather Murray, Todd Machemer and 8 more

Open access · goldAbstract readClinical Trial, Phase IRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in ESC heart failure, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 57 papers.

0numbers the graph read from it
0cells of the map it votes in
57citing papers in PubMed
3.3field-weighted citation impact, top 6% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

57 citing papers in PubMed, 86 citations in OpenAlex.

  1. Trial
  2. Trial
  3. Trial
  4. Review
  5. Article
  6. Biologics for cardiovascular diseases: from bench to bedside.Signal transduction and targeted therapy · 2026
    Review
  7. Review
  8. Antisense Oligonucleotide Therapeutics Targeting Age-Related Diseases.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2026
    Review
  9. Review
  10. Designing nucleic acid-based therapeutics for cancer treatment: Updates on the state of the art.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Review
  11. Article
  12. Eplontersen for the Treatment of Hereditary Transthyretin Amyloidosis.Methods in molecular biology (Clifton, N.J.) · 2026
    Article
  13. Tissue Pharmacokinetics of Antisense Oligonucleotides.Methods in molecular biology (Clifton, N.J.) · 2026
    Article
  14. Review
  15. Review
  16. Review
  17. Review
  18. Review
  19. Review
  20. Cardiac amyloidosis: Innovations in diagnosis and treatment.European heart journal supplements : journal of the European Society of Cardiology · 2025
    Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

18 authors at 2 institutions in 1 country.

Nicholas J VineyIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Shuling GuoIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Li-Jung TaiIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Brenda F BakerIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Mariam AghajanIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Shiangtung W JungIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Rosie Z YuIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Sheri BootenIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Heather MurrayIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Todd MachemerIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Sebastien BurelIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Sue MurrayIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Gustavo BucheleIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Sotirios TsimikasIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Eugene SchneiderIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Richard S GearyIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Merrill D BensonIndiana University School of Medicine, Indianapolis, IN, USA.
Brett P MoniaIonis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA, 92010, USA.
Ionis Pharmaceuticals (United States) · USIndiana University School of Medicine

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

aimsAmyloidogenic transthyretin (ATTR) amyloidosis is a fatal disease characterized by progressive cardiomyopathy and/or polyneuropathy. AKCEA-TTR-L METHODS AND

resultsAKCEA-TTR-L

conclusionsThese findings suggest an improved safety and tolerability profile with the increase in potency achieved by productive receptor-mediated uptake of AKCEA-TTR-L

Indexed as

Amyloid Neuropathies, FamilialOligonucleotides, AntisenseAnimalsHealthy VolunteersHumansLigandsMicePrealbuminLigandsOligonucleotides, AntisensePrealbuminAmyloidosisAntisenseCardiomyopathyLigand-conjugatedPolyneuropathyTransthyretin

Identifiers

PMID33283485
PMCPMC7835591
OpenAlexW3111860220

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.